Dr. Fischer @ Children’s Hospital Los Angeles

Dr. Fischer is an expert in genetic therapy.
My first message to Dr. Fischer was in October 23, 2001, he answered a day later October 24, 2001.
I found this article about a speech he gave at Children’s Hospital Los Angeles and it has interesting information.
Source: Childrens Hospital Los Angeles
“If you want to better treat a given disease — in our context, a primary immunodeficiency — it’s first absolutely crucial to understand as much as we can about the mechanism of the disease,” explained Dr. Fischer. “So, for genetic disorders obviously it means to identify the gene -mutations of which caused the disease — and then to understand what the protein encoded by the gene normally does — what’s its role — so then we can start to think about potential therapeutics.”
“One possibility for some of these diseases is the application of gene therapy,” he said, “but this is fully based on our understanding the mechanisms of the disease. Then, of course, one should design the best way to add a normal copy of the gene into the cells which are abnormal. In the case of the diseases we are interested in — the immune deficiencies — these are the progenitors in the bone marrow which give rise to different kinds of lymphocytes.”
He discussed approaches that have been used to treat these diseases over the years, such as bone marrow transplant, which has restored life to many but not all of these patients. Recent studies show that in some of the children who are 10 or more years post-transplant, the effect is being lost and they are becoming immune deficient again. This late loss of protective immunity may occur because they received the more “grown-up” cells that made T-cells for awhile, but they did not receive sufficient stem cells that can make T-cells for decades.
Read the complete article here.

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